Friday, May 27, 2011

On May 24, CHI hosted an insightful briefing in Washington, D.C. highlighting the importance of therapeutic advancements and innovations in musculoskeletal disease and injuries. The program was held on Capitol Hill, and was attended by congressional staff, patient advocates, industry representatives and members of the press, among others. The assembled panel of experts addressed a variety of issues in the area of musculoskeletal conditions, providing an excellent awareness-raising overview and highlighting some of the successful research, technologies, and treatments being offered by CHI members.

Todd Gillenwater, CHI’s senior vice president of public policy, opened the program by detailing the impact of musculoskeletal disease in the United States. In particular, he noted that in the U.S. alone, these conditions are a leading cause of disability, accounting for more than 130 million patient visits to healthcare providers each year. Further, he said the annual cost of treatment associated with musculoskeletal disease is estimated at a staggering $254 billion.

Then, CHI’s invited panel of experts took the podium in turn to present their own unique perspectives on musculoskeletal health. First to the podium was Dr. Said Ibrahim, professor of medicine and vice chair of the Department of Medicine at the University of Pennsylvania School of Medicine. His presentation focused on health disparity research in the area of joint replacement procedures, which has shown that minorities, African Americans in particular, are less likely than Caucasians to opt for elective joint replacement procedures. His findings, adjusted for key factors such as insurance coverage, indicated that the disparity was related to the fact that African Americans were less familiar with the procedures and more concerned about things like post-operative pain and recovery time. Ibrahim said he believes that this disparity is now well enough understood to be effectively addressed by a targeted educational campaign.

Dr. Deborah Kado, associate professor with the Departments of Orthopedic Surgery and Medicine at the Geffen School of Medicine at UCLA, presented research on hyperkyphosis — a musculoskeletal aging condition that creates humped necks or upper backs in men and women. The condition is now recognized as a geriatric syndrome, yet surprisingly little is known about its causes or potential means of prevention. Preliminary studies, including Kado’s own National Institutes of Health-funded projects, have demonstrated that the condition decreases lung function and quality of life, while increasing rates of falls, injury and mortality. Kado’s team has received renewed NIH funding to continue its work on hyperkyphosis through a 1,800-member cohort study of men over the age of 65.

Next to the podium was Laetitia Cousin, vice president of regulatory and clinical affairs and quality assurance with San Diego-based NuVasive. Cousin discussed NuVasive’s explosive growth led by their innovative, minimally disruptive spinal surgery devices and techniques. NuVasive’s next-generation surgical methods and tools make their procedure’s quality-adjusted life year (QALY) outcomes equal to or better than that of hip surgery, helping to erode spinal surgery’s poor 1990s-era reputation. Cousin closed by laying out two main obstacles to NuVasive’s willingness and ability to reinvest its recent profits in additional R&D and growth: U.S. Food and Drug Administration clearance/approval delays and the medical device excise tax. NuVasive recommended improved FDA transparency through guidance documents; improved FDA reviewer retention and expertise through training; improved FDA review process efficiency through earlier and more frequent collaboration with industry; and repeal of the device tax.

Last to present was Scott Simonet, Ph.D, vice president of research for Amgen. Simonet described the 15-year process to discover and develop Amgen’s denosumab, a drug that regulates bone density and helps the human body limit bone reabsorbtion. With precursor science first discovered in 1995, the product received FDA and European regulatory approval to treat osteoporosis in 2010, as well as FDA approval for cancer-related indications in the same year. While Simonet was sparing in his willingness to express complaints associated with denosumab’s approval process, he did make a point to show the length and complexity of the product’s discovery and development chain.

The presentations spurred a lively and candid follow-on discussion between the panelists and the audience. As the event closed, many expressed their thanks to CHI for organizing the session, with more than one attendee saying that that they had found it “very educational.”

Thursday, May 26, 2011

LoneStar Heart got its start in Southern California as CardioPolymers, with the goal of changing the way we treat heart failure and other cardiac abnormalities. Frank Ahmann, a veteran of the biotech and medical device industries, brought his expertise to LoneStar Heart following a management career in Europe and North America at Baxter International’s biotech and cardiovascular group, now Edwards LifeSciences. Among many products, he launched the first comprehensive system for the collection, processing, storage, and administration of hematopoietic stem cells. A German citizen who grew up in Mexico, Ahmann has strong ties to the European medical market and speaks English, Spanish and German. He holds a bachelor’s degree from the University of Texas at Austin and a master’s in business administration from Columbia University Graduate School of Business.Q: Tell me something that makes LoneStar Heart unique.

A: We are not a typical venture capital-funded company. We have been funded privately by a very small group of wealthy individuals based in Texas. In the future, we expect to fund our growth through corporate partnerships and investments as well as the continued participation of our private investors.

Q: Talk about the technologies that originated at University of California San Francisco and led to the foundation of LoneStar Heart.

A: The original idea before LoneStar Heart was even formed was a company by the name of CardioPolymers Inc., based here in Southern California with technology from the University of California San Francisco (UCSF) and a gentleman by the name of Dr. Randall Lee, who was an electrophysiologist and professor there.

He was looking at a way to use an implantable hydrogel in combination with stem cells or growth factors. One of the shortcomings of stem cell therapies is that they don’t linger in the heart muscle very long or at all, and the effect of stem cell therapy and growth factor therapies is very, very limited and lasts for a very short time. So, nobody has really been able to show in significant clinical studies that stem cells in the heart regenerate tissue and improve long-term and permanently the condition of the failing heart.

The issue became “Can we make this better? Can we find a way for these stem cells to actually stay in the heart?” The one way to do that is to mix in these stem cells with the hydrogel.

Q: Have hydrogels been used in any other stem cell applications before?

A: Not in the heart at all. It is completely novel for the heart. As we were testing it, we found out that the hydrogel, without stem cells, had a very significant and positive effect on improving the mechanics of the heart, which led to the product that it is today: Algisyl. This product is essentially a gel that is derived from brown algae. It is extremely purified to remove all endotoxins and make it inert. It is liquid when you inject it and then it solidifies to the right degree when it is in the heart.

Normally, what you would expect is that the material will degrade after a few weeks. In our case, the material stayed put in preclinical models. After two years, the hydrogels were still there, in the same quantity and in the same position as they were in the beginning.

Suddenly, we found ourselves with a long-term, implantable device. It is a device because it has no pharmacologic or immunologic action.

Q: So how might this be useful in congestive heart failure?

A: A large majority of patients that have heart failure have a dilated, stretched out heart muscle that has a decreasing pumping efficiency and is incapable of delivering enough blood to the entire body. This is a vicious circle that continues to get worse with time and the implantable hydrogel actually stops or even reverses this negative trend. It accomplishes this by reducing the tension and the stress to the heart muscle purely through mechanical means.

Beyond this first step, to be able to achieve true regeneration of tissue and provide even greater efficacy, you will most likely have to combine a couple approaches. So, our idea has been all along to have the implantable hydrogel, which acts to mechanically improve the heart, combined with bioreactive small molecules or biologics that we have licensed from the University of Texas to provide the ability for tissue regeneration.

Q: What are you noticing in your initial studies?

A: Up to this point, we have treated a total of 11 patients — a majority of them are longer than one year out from when they received the implant. The safety, feasibility, and initial signals of efficacy look very positive. Therefore, we will soon begin a controlled randomized study in Europe to show efficacy delivering the product through surgical techniques. Later, an interventional catheter-based delivery will be added to the product mix.

We expect that in two years we will have enough data to receive approval to market in the European Union with the first version of the product.

Wednesday, May 25, 2011

Record-breaking attendance of more than 600 VCs and entrepreneurs convened for OCTANe’s Annual VC in the OC event, May 24, in Irvine, Calif. OCTANe CEO Matthew Jenusaitis kicked off with highlights from the OC Innovation Report, demonstrating that by quantitative and qualitative measures on the state of innovation, overall environment and investment, Orange County is an epicenter for medical technology and high tech in the U.S.

“Don’t overlook alliance opportunities with middle-size companies and consider the advantages of these compared to larger companies to open up the range of potential partners,” Stevenson advises.

“As a company, alliances are critical to Allergan to keep our growth rate of 12-15 percent per year," said Pal. "We are looking at technologies in all stages of development, whether early-stage or late-stage commercialization."

Kehl weighed in on Edwards Lifesciences investment in R&D: “Edwards will be flexible in structuring the deal, but the technology must be centrally relevant to our business. In the current environment, it is taking more and more to get product to market, so the R&D line is sacred and will grow through alliances.” Check out Deloitte’s study: "Critical Factors for Alliance Formation."

Mark Stevens, Partner at Sequoia Capital gave the lunch keynote. He described he and his wife Mary’s vision in creating the USC Stevens Institute for Innovation with a $200 million donation to develop a university-wide resource for USC students and faculty to transition beyond concepts into real-world implementation and address untapped opportunities. As far as new sources of start-up capital, Stevens recommends entrepreneurs access microfunds. Microfunds are typically run by high-wealth individuals with industry-specific experience who are very hands-on, investing smaller amounts and combining that with their bandwidth and connections to shepherd the company through early-stage development.

Don Milder, partner with Versant Ventures, provided his perspective on healthcare investing on the OC VC panel. He sees the U.S. Food and Drug Administration improving but still in need of reform.

"Never underestimate the power of one transforming a bureaucracy," he said. "That one individual who is making strides to improve FDA for all of us in med tech is Josh Makower."

Monday, May 23, 2011

CHI and Hogan Lovells convened a half-day program on the U.S. Food and Drug Administration (FDA) drug lag and the challenges and opportunities in working with the agency on new drug applications. Our guests heard from speakers including CHI’s CEO David Gollaher, Ph.D.; Rick Winningham, chief executive officer of Theravance Inc.; Marcea Lloyd, senior vice president, government and corporate affairs and general counsel at Amylin Pharmaceuticals; Beth Seidenberg, M.D., partner with Kleiner Perkins Caufield & Byers; Joseph T. Kennedy, vice president, general counsel and secretary for Transcept Pharmaceuticals, Shelley Chu, M.D., Ph.D. principal of Frazier Healthdcare; and Hogan Lovells partners Robert Church, Kevin Clayton, David Fox and Lynn Mehler.

Gollaher opened the proceedings in his keynote address, by providing highlights from CHI’s recent publication, Competitiveness and Regulation: The FDA and the Future of America’s Biomedical Industry. According to the report, which uses FDA’s own data to establish the relationship between regulation and the competitiveness of the industry, today’s FDA is not keeping pace with the innovation being produced here. The relationship between FDA and industry is strained by unexplained regulatory delays, a lack of clear standards for clinical data and an inconsistent and unpredictable pattern of communication, which has led to an increase in risk and a crisis in biomedical R&D funding. The resulting flight of product launches to EU countries should be cause for concern for everyone from policymakers to the patients who are most in need.

Subsequent speakers, including Lloyd and Winningham provided insights as executives from biomedical companies with products either currently in review at FDA or on the market, into the struggles these innovators are facing, with the capital markets in crisis and an uncertain regulatory process. Key takeaways from these people on the front lines included:

This business is about patients. Period.

Success depends upon taking, recognizing and managing risk.

Surround yourself with smart advisors and even smarter employees.

Precise communication is key – among all your trusted advisors and employees involved in the clinical trials, data analysis and regulatory approval of products, and regulators.

It’s not just about strategy…perfect execution is key.

Only the smart and persistent will survive in this climate.

More companies will be forced to specialize and put all resources toward the most successful projects due to the higher barrier to entry.

Dr. Seidenberg, partner with Kleiner Perkins Caufield & Byers and Dr. Chu, principal with Frazier Healthcare, provided valuable insights from the venture capital perspective, explaining the consequences of increased risk, including money and people going overseas, not just to the EU, but to China. High tech and life sciences are both catalysts for growth overseas and Seidenberg noted that foreign executives are coming here to get educated and going back to their homelands to research, develop and commercialize medical technologies that have greater chance of being commercialized sooner overseas, rather than in the US. The regulatory and business environment in the US is “absolutely influencing our investing,” said Seidenberg, “we can’t sit back and let the US lose its competitive edge.”

When panelists, including Hogan Lovells experts from their global life sciences practice, Church, Clayton, Fox and Mehler, were asked what we can do to stem this tide of medical innovation leaving the U.S. in search of friendlier tax and regulatory environments, here were some of the insights shared:

We need more dialogue between industry and regulators.

We need clarification on the decision-making process, timelines and goals.

We need more expert reviewers at FDA.

We need to open the lens to assess risk and benefit in a systemic way.

Without real, substantive change, the pain is going to go beyond investors and innovators – this is a crisis that is going to hurt everybody – most notably, the patients who need innovative, world-class therapies.

Stay tuned, as CHI and Hogan Lovells plan to release an Executive Summary on the proceedings from the meeting. For more information, please call CHI at (858) 551-6677.

Thursday, May 5, 2011

﻿Scott Johnson was only 20 years old when he was diagnosed with multiple sclerosis. In 2002, after waiting 26 years for a cure, Johnson took things into his own hands and leveraged his background as a business consultant and serial entrepreneur to create the Accelerated Research Collaboration model to reform the medical research and drug development system that has failed to produce desperately needed new patient treatments for millions living with chronic and debilitating diseases. In 2006, Johnson was named as one of Scientific American magazine's top 50 worldwide for business, science and policy leadership. In 2010, he received a Northern California Ernst & Young Entrepreneur of the Year Award. In 2011, Fast Company magazine named the Myelin Repair Foundation one of the top 10 biotech companies.

Q: What was the impetus of starting MRF?

A: In 1976, at the age of 20, I was diagnosed with multiple sclerosis. Unlike many who are diagnosed with MS, I was fortunate to be able to pursue an active business career, including leading three start-up companies. In 2001, I read a news brief in BusinessWeek that described work being done at Yale on myelin repair in MS. (Myelin is the insulating substance coating the nerves in the brain and spinal cord. It is also the substance that is damaged in MS. This damage is what causes the disease’s unpredictable symptoms). Up to that point, everything I had read about MS had focused on how to cure the disease or how to slow the immune reaction. So I picked up the phone, tracked the scientist down and started asking questions.

I learned two important lessons from my conversation with this scientist and others. First, scientists were starting to believe that regeneration or repair in the central nervous system was possible, and that it probably happens naturally in healthy people. Second, and perhaps the real lesson, was that it seemed that if you did things slightly differently throughout the entire continuum of medical research and drug discovery that the process of developing new medicines could be greatly accelerated. That was really the genesis of the Myelin Repair Foundation.

Q: What is the Accelerated Research Collaboration model and how is it different from traditional approaches to basic research and drug discovery?

A: The traditional model for medical research and drug discovery typically divides into several parts: basic biology, often conducted in an academic laboratory; pharmaceutical drug development; clinical trials; and FDA approval. For a variety of reasons, over the past several decades, these entities have grown farther and farther apart. The result has been a failure, essentially, to connect the vital dots that would lead to bringing a new medicine to market. That is why, in spite of a doubling of investment in research from 1995-2005, the number of new drug approvals has remained flat.

The Accelerated Research Collaboration model optimizes and integrates the contributions of all those who participate in the value chain of making new medicines. As a result, it provides a larger number of therapeutically relevant treatment targets in the pipeline, it translates discoveries made in animal models to the human system, it builds partnerships with pharmaceutical companies to develop and bring new treatments to market, and, ultimately, improves quality of life for millions living with diseases for which there are currently no effective treatment or cures.

Q: What are some of the exciting, new initiatives the MRF will be launching in the next 1-5 years?

A: The success of our discovery biology program has brought us many high-quality therapeutic targets and several new research tools that may be used more broadly in research for other neurological diseases.

At this time, we have two development paths before us: to validate the most promising novel myelin repair therapeutic targets through contract research organizations and to investigate the potential for repositioning of more than 40 targets that are in clinical development by various companies for other indications. We expect to pursue these paths in parallel and have developed a robust translational medicine platform to accomplish this.

We are also investing in the development of myelin repair biomarkers. Establishing one or more clearly measurable biomarkers will help us select the most promising of our targets for development.

Q: How are you measuring success in the near and long term?

A: Near-term success is already being measured by the number of targets identified in our funded laboratories as well as the number of new research tools that are enabling this research. In the longer term, of course, the only real measure of success will be one or more successful clinical trials for a myelin repair therapy. We are currently aiming for a myelin repair clinical trial for one of our targets in 2014.

Q: What is your fundraising strategy?

A: Though many think of us as a biotech company, we are in fact a non-profit organization supported by generous individuals, corporations and foundations with either an interest in finding a cure for MS or an interest in improving the outcomes of all medical research. In six and a half years, we have raised gifts and pledges totaling $43 million. We anticipate the next phase of our work will require as much as $80 million to complete.

Q: What is your partnership strategy?

A: There has been considerable interest from several companies in our both our portfolio of targets and our research tools. At this time, we are seeking partnerships with pharmaceutical companies who have an interest in myelin repair or who have a compound in clinical development for another disease indication that from our research appears to affect the same mechanisms for myelin repair. We are reaching gout to those companies now.